22 Clinical Trial Alert_1024x5123

Clinical Study Alert: A Phase 1/2 Study of Safety and Efficacy of LX2006 Gene Therapy in Adults with Cardiomyopathy Associated with Friedreich Ataxia

Researchers at Lexeo Therapeutics, Inc. (Lexeo) are seeking adults with cardiomyopathy associated with Friedreich ataxia (FRDA) to participate in an open label, dose-ascending, multicenter Phase 1/2 clinical trial, called SUNRISE-FA (NCT# 05445323) to evaluate the safety and efficacy of the investigational gene therapy LX2006. FRDA is caused by mutations in the FXN gene that lead to the loss of frataxin, an essential protein for the nervous system and heart to produce energy and work properly. LX2006 is designed to deliver a functional copy of the FXN gene to heart cells to enable them to produce more frataxin protein.

All study participants will receive one dose of LX2006 investigational treatment. This study will last approximately 5.5 years in total (inclusive of a screening period that may take up to 5 months, a 52-week study assessment period, and 4-year long-term follow-up period). Following the screening period, participants will be required to attend 15 office visits during the first year of the study, and then five study center visits and three remote visits during the long-term follow-up period.

The drug will be administered intravenously (in the vein) only once. The effects of LX2006 will be evaluated using a number of tests and procedures at each visit including but not limited to: medical history, physical examination, vital signs, laboratory tests (including blood draws), heart biopsy, chest X-ray, liver ultrasound, heart function tests, and FA questionnaires and assessments.

To be eligible, individuals must meet specific  inclusion criteria, including:

  • Individuals aged 18 to 50 years old
  • Confirmed genetic diagnosis of FRDA with onset being before 25 years of age
  • Evidence of cardiomyopathy based on specific criteria

Individuals may not be eligible to participate if they are affected by another condition or receiving another treatment that might interfere with the ability to undergo safe testing.

Additional considerations for participation include the following:

  • Participants already taking Skyclarys® (omaveloxolone) can remain on the medication but must be on a stable dose for a minimum of 12-weeks prior to screening.
  • If an individual is not on omaveloxolone at the time of screening, they have the option to start taking the medication one year after LX2006 administration.

Full inclusion and exclusion criteria may be provided upon request.  Eligibility will be evaluated by the clinical trial doctor to confirm participation in the study. Please visit this link for additional information on the clinical trial including active trial sites.

The investigational gene therapy and all trial-related assessments will be provided at no cost to study participants. Any reasonable trial-related expenses such as fees for parking, meals, will also be covered by the study sponsor, Lexeo. Travel and accommodation will be arranged for participants in the trial and one caregiver.

Taking part in this trial is voluntary. Once a participant enrolls in the trial, they can leave the trial at any time, for any reason.

Safety and efficacy have not been established. LX2006 has not been approved by the U.S. Food and Drug Administration or any other country’s health authority or regulatory agency. There is no guarantee that an individual will receive any benefits and there may be risks and side effects associated with taking part in this study.

To learn more about the study or inquire about participation, contact the Lexeo team at email: clinicaltrials@lexeotx.com.

MDA does not endorse any brands, services or products, and this does not constitute an endorsement by MDA. MDA makes this information available for informational purposes only.

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